Upfront allogeneic transplantation versus JAK inhibitor therapy for patients with myelofibrosis: a North American collaborative study

Dawn Maze; Murat O Arcasoy; Ryan Henrie; Sonia Cerquozzi; Rammurti Kamble; Samer Al-Hadidi; Abdulraheem Yacoub; Anurag K Singh; Mahmoud Elsawy; Shireen Sirhan; Elliot Smith; Curtis Marcoux; Auro Viswabandya; Andrew Daly; Hassan Sibai; Caroline McNamara; Yuliang Shi; Wei Xu; Katherine Lajkosz; Lynda Foltz; Vikas Gupta

doi:10.1038/s41409-023-02146-6

Upfront allogeneic transplantation versus JAK inhibitor therapy for patients with myelofibrosis: a North American collaborative study

Bone Marrow Transplant. 2024 Feb;59(2):196-202. doi: 10.1038/s41409-023-02146-6. Epub 2023 Nov 8.

Authors

Dawn Maze¹, Murat O Arcasoy², Ryan Henrie³, Sonia Cerquozzi⁴, Rammurti Kamble⁵, Samer Al-Hadidi⁶, Abdulraheem Yacoub⁷, Anurag K Singh⁷, Mahmoud Elsawy⁸, Shireen Sirhan⁹, Elliot Smith¹⁰, Curtis Marcoux⁸, Auro Viswabandya¹⁰, Andrew Daly⁴, Hassan Sibai¹⁰, Caroline McNamara¹⁰, Yuliang Shi^{11

12}, Wei Xu^{11

13}, Katherine Lajkosz^{11

13}, Lynda Foltz³, Vikas Gupta¹⁰

Affiliations

¹ The Princess Margaret Cancer Centre, University of Toronto, Toronto, ON, Canada. dawn.maze@uhn.ca.
² Duke Cancer Institute, Duke University School of Medicine, Durham, NC, USA.
³ Division of Hematology, St. Paul's Hospital, University of British Columbia, Vancouver, BC, Canada.
⁴ Tom Baker Cancer Centre, Alberta Health Service Calgary Zone, University of Calgary, Calgary, AB, Canada.
⁵ Center for Cell and Gene Therapy, Baylor College of Medicine and Houston Methodist Hospital, Houston, TX, USA.
⁶ Myeloma Section, Winthrop P. Rockefeller Cancer Institute, University of Arkansas for Medical Sciences, Little Rock, AR, USA.
⁷ Division of Hematology and Oncology, University of Kansas Medical Center, Kansas City, KS, USA.
⁸ Division of Hematology, Dalhousie University, Halifax, NS, Canada.
⁹ Jewish General Hospital, McGill University, Montreal, QC, Canada.
¹⁰ The Princess Margaret Cancer Centre, University of Toronto, Toronto, ON, Canada.
¹¹ Department of Biostatistics, Princess Margaret Cancer Centre, University Health Network, Toronto, ON, Canada.
¹² Department of Statistics and Actuarial Science, University of Waterloo, Waterloo, ON, Canada.
¹³ Dalla Lana School of Public Health, University of Toronto, Toronto, ON, Canada.

Abstract

Allogeneic hematopoietic cell transplantation (HCT) is the only curative therapy for myelofibrosis (MF) and is recommended for patients with higher risk disease. However, there is a risk of early mortality, and optimal timing is unknown. JAK inhibitor (JAKi) therapy may offer durable improvement in symptoms, splenomegaly and quality of life. The aim of this multicentre, retrospective observational study was to compare outcomes of patients aged 70 years or below with MF in chronic phase who received upfront JAKi therapy vs. upfront HCT in dynamic international prognostic scoring system (DIPSS)-stratified categories. For the whole study cohort, median overall survival (OS) was longer for patients who received a JAKi vs. upfront HCT, 69 (95% CI 57-89) vs. 42 (95% CI 20-not reached, NR) months, respectively (p = 0.01). In patients with intermediate-2 and high-risk disease, median OS was 55 (95% CI 36-73) months with JAKi vs. 36 (95% CI 20-NR) months for HCT (p = 0.27). An upfront HCT strategy was associated with early mortality and difference in median OS was not observed in any risk group by 5 years of follow-up. Within the limitations of a retrospective observational study, we did not observe any benefit of a universal upfront HCT approach for higher-risk MF.

Publication types

Observational Study
Multicenter Study

MeSH terms

Hematopoietic Stem Cell Transplantation* / adverse effects
Humans
Janus Kinase Inhibitors*
North America
Primary Myelofibrosis*
Quality of Life
Retrospective Studies
Transplantation, Homologous

Substances

Janus Kinase Inhibitors